NHS to offer £1.8m drug to cure babies of spinal disease
The NHS will offer a £1.8 million drug to cure babies of a spinal disease that can kill babies before they are two years old
- Around 65 babies born each year in England have spinal muscular atrophy
- The disease causes muscle weakness, paralysis, and breathing problems.
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Babies born with a genetic disease that leads to a life expectancy of just two years without intervention will now be cured on the NHS with a new £1.8m drug.
Babies with severe spinal muscular atrophy (SMA), which causes muscle weakness and leads to paralysis and breathing problems, will now be able to take the gene therapy drug Zolgensma.
Treatment surveillance The National Institute for Health and Care Excellence (Nice) has approved the use of onasemnogene abeparvovec, better known by its trade name.
Around 65 babies born each year in England have spinal muscular atrophy, which is the leading genetic cause of death in children.
The disease causes a defect in a gene called SMN1, which makes an essential protein and therefore allows spinal nerves in the spinal cord to control muscle movement.
Arthur Morgan, then five months old, with his father Reece Morgan in June 2021 after becoming the first patient in England to undergo Zolgensma gene therapy.
The most serious form of the condition, SMA type 1, can develop in babies younger than six months, with a 90% chance of dying before the age of two if left untreated. The times reports.
But Zolgensma has been shown in studies to help patients sit up, crawl and walk, something they would never normally be able to do, and it also prevents them from being put on a ventilator.
The single infusion, administered in just one hour, uses a harmless virus to deliver a healthy version of the SMN1 gene.
It goes into the nerve cells to replace the faulty gene and restore normal function, allowing the baby to live a healthy life with minimal symptoms.
It comes after year-old Edward Willis-Hall was given the drug and subsequently took his first assisted steps in January earlier this year.
His mother, Megan Willis, 30, spoke of her joy after her son achieved milestones she never thought possible.
He can turn around, sit down, stand for five minutes in leggings that support his legs, and take steps in a harness.
She told the BBC: ‘We are very proud of Edward. He is doing amazingly well. He is far exceeding our expectations. We are so incredibly lucky.
Ms. Willis, who was told her son had spinal muscular atrophy when he was seven weeks old, set a goal for him to sit up without assistance. She said: “All I ever wanted was for him to be able to sit back and I knew he would then have an amazing life.”
Nice has said buying the drug is a “profitable” use of resources.
Currently, only babies with a family history of SMA will be tested.
The single infusion, administered in just one hour, uses a harmless virus to deliver a healthy version of the SMN1 gene.
But activists are pushing for all five-day-old babies to undergo screening for nine conditions.
Laurent Servais, professor of pediatric neuromuscular diseases at the University of Oxford, said: “I have seen too many families devastated by this disease, but now we have treatment options.”
‘However, waiting until the onset of symptoms is still too late. Every day we delay in finding and treating these babies, we could be responsible for a child spending her life in a wheelchair.
“There is 100 per cent consensus among UK experts, among global experts, among patients: we need to test to save lives, we need to test to save quality of life, and we need to test to save money.”
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